Ipsen’s IPN60340 receives FDA Breakthrough Therapy Designation for first-line unfit AML
Ipsen has announced that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation (BTD) to IPN60340 in combination with venetoclax and azacitidine for the first-line treatment of patients with acute myeloid leukaemia (AML) who are not suitable for intensive chemotherapy.
IPN60340 is an investigational, first-in-class monoclonal antibody targeting BTN3A, an immune-regulatory protein broadly expressed across solid and blood cancers.
The Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions where early clinical data indicate a potential substantial improvement over existing therapies.
IPN60340 was previously granted Orphan Drug Designation by both the U.S. FDA and the European Medicines Agency in July 2025.
The designation is based on results from the Phase I/II EVICTION study. Updated data showed that IPN60340 in combination with venetoclax and azacitidine produced high response rates in newly diagnosed, unfit AML patients, with complete response rates close to double those seen in historical standard-of-care data across multiple molecular subtypes.
Ipsen plans to engage with the FDA in the first half of 2026 to discuss Phase II/III development plans for IPN60340 in AML.
